Doxycycline Injection for Neurofibromatosis Type 2

Overseen By Christopher Wood

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Massachusetts Eye and Ear Infirmary

Must not be taking: Anticancer therapies, Glucocorticoids

Disqualifiers: Allergy to doxycycline, Unstable disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 4

Duration: 1 day

19 Participants Needed

Trial Summary

What is the purpose of this trial?

In this research study the investigators want to learn more about an alternate, local treatment for skin schwannomas. Specifically, local doxycycline intra-tumoral injection will be performed as a potential treatment for NF2-related skin schwannomas, ultimately reducing the risks and costs associated with standard surgical removal of such skin tumors if successful.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are currently receiving anticancer therapies or have had them within 4 weeks before the study. It's best to discuss your specific medications with the study team.

What data supports the effectiveness of the drug doxycycline injection for treating Neurofibromatosis Type 2?

Research shows that doxycycline can enhance the effectiveness of certain cancer treatments by reducing tumor growth and increasing the death of cancer cells. This suggests that doxycycline might help in treating tumors associated with Neurofibromatosis Type 2 by potentially reducing tumor size and growth. ^{1 2 3 4 5}

Is doxycycline injection safe for humans?

Doxycycline has been used safely in humans for various conditions, including resolving skin issues caused by other medications, as seen in a 12-year-old girl with neurofibromatosis type 1. This suggests it is generally safe, but specific safety data for doxycycline injection in neurofibromatosis type 2 is not provided. ^{6 7 8 9 10}

How does the drug Doxycycline Injection differ from other treatments for neurofibromatosis type 2?

Doxycycline Injection is unique because it is being explored as a treatment option for neurofibromatosis type 2, a condition with limited systemic treatments. Unlike the commonly used drug bevacizumab, which requires long-term use and can lead to tumor regrowth after stopping, doxycycline's potential benefits and mechanism in NF2 are still under investigation, offering a novel approach. ^{6 8 11 12 13}

Research Team

D. Bradley Welling, MD, PhD

Principal Investigator

Massachusetts Eye and Ear Infirmary

Eligibility Criteria

This trial is for individuals aged 8 or older with neurofibromatosis type 2 (NF2) and skin schwannomas. Participants must have stable neurological conditions, a life expectancy over a year, and be able to give consent. They should not be on recent anticancer treatments or have severe medical issues that could affect participation.

Inclusion Criteria

I am 8 years old or older.

I have no lingering side effects from previous cancer treatments.

My neurological symptoms have been stable for at least a week.

Exclusion Criteria

My tumor is on my face or affects major nerves for movement.

I haven't had radiation on the tumor I want to study in the last year or any radiation in the last 4 weeks.

I am not pregnant, breastfeeding, and if capable of having children, I am using effective birth control.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive local doxycycline intra-tumoral injection for NF2-related skin schwannomas

1 day

1 visit (in-person)

Follow-up

Participants are monitored for changes in tumor size and other symptoms

6 months

2 visits (in-person)

Long-term Follow-up

Participants are monitored for changes in tumor size and other symptoms

1 year

1 visit (in-person)

Treatment Details

Interventions

• Doxycycline Injection

Trial Overview The study tests local injections of Doxycycline directly into the skin tumors of NF2 patients as an alternative to surgery. The goal is to see if this method can effectively reduce tumor size while being less risky and costly than standard surgical procedures.

Participant Groups

Group I: Single Arm Open Label Experimental Treatment 1 Intervention

Open Label

Who Is Running the Clinical Trial?

Massachusetts Eye and Ear Infirmary

Lead Sponsor

Trials

115

Recruited

15,000+

Findings from Research

Topical photodynamic therapy (PDT) using aminolevulinic acid (ALA) was shown to effectively target cutaneous neurofibromas (cNF) in a phase I trial with 27 participants, demonstrating significant fluorescence in tumors and minimal impact on surrounding normal skin.

The treatment resulted in a notable increase in apoptotic cells in ALA-treated tumors compared to vehicle-treated tumors, indicating that PDT may provide a less invasive and tissue-sparing option for managing neurofibromatosis type 1.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. Quirk, B., Olasz, E., Kumar, S., et al.[2021]

References

1. United States pubmed.ncbi.nlm.nih.gov

Novel use of doxycycline in continuous-instillation negative pressure wound therapy as "wound chemotherapy". [2013]

2. England pubmed.ncbi.nlm.nih.gov

Doxycycline influences microcirculation patterns in B16 melanoma. [2013]

3. United States pubmed.ncbi.nlm.nih.gov

Doxycycline potentiates antitumor effect of 5-aminolevulinic acid-mediated photodynamic therapy in malignant peripheral nerve sheath tumor cells. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Periocular neurofibrosarcoma in a horse. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Treatment of MEK inhibitor-induced paronychia with doxycycline. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Target Product Profile for Cutaneous Neurofibromas: Clinical Trials to Prevent, Arrest, or Regress Cutaneous Neurofibromas. [2023]

8. India pubmed.ncbi.nlm.nih.gov

A Safe Alternative in Neurofibromatosis for Lower Limb Surgeries: Combined Femoral and Sciatic Nerve Block. [2020]

9. United States pubmed.ncbi.nlm.nih.gov

Management of Central and Peripheral Nervous System Tumors in Patients with Neurofibromatosis. [2023]

10. United States pubmed.ncbi.nlm.nih.gov

Subsequent Neoplasms After a Primary Tumor in Individuals With Neurofibromatosis Type 1. [2021]

11. England pubmed.ncbi.nlm.nih.gov

Treatment of neurofibromatosis type II with anlotinib: a case report and literature review. [2023]

12. England pubmed.ncbi.nlm.nih.gov

Long-term therapy with Bevacizumab in a young patient affected by NF2. Stop or continue treatment? An update of a case report and review of the literature. [2021]

13. Switzerland pubmed.ncbi.nlm.nih.gov

Treatment of neurofibromas with a carbon dioxide laser: a retrospective cross-sectional study of 106 patients. [2015]